Amicus Therapeutics

Encouraging results have been reported for the first three dosage groups in a phase 2 trial of the 'pharmacological chaperone' AT2220

posted on October 12, 2012 - 3:21pm

Biopharmaceutical company Amicus Therapeutics presented updated and encouraging results for its experimental Pompe disease (acid maltase deficiency) compound AT2220 this week at the 17th International Congress of the World Muscle Society in Perth, Australia.

Most anybody who hears John Crowley’s story will call him a hero.
It’s a logical label for a man who led a quest to develop a treatment to stop the disease killing two of his young children.
The Crowley family’s incredible story has been chronicled in the media, a book and now a major Hollywood feature film. “Extraordinary Measures” will be released Jan. 22 with an A-list cast that includes...

John Crowley is the father of two children with Pompe disease (acid maltase deficiency). His quest to develop a drug to save their lives is chronicled in the movie "Extraordinary Measures," to be released by CBS Films in January 2010.
Crowley was interviewed for the Quest article "Heroes, Hope & Hollywood." Below are more of his thoughts on the "golden age" of drug development and how...

The approval of Myozyme in 2006, after decades of research, has transformed Pompe disease from a severely disabling or fatal condition into a treatable, chronic disorder. But responses to the drug vary, its cost is high, and some questions remain unanswered.

Mia Hanley of Cranston, R.I., looks pretty much like any 4-year-old preschooler. She’s a normal height and weight, walks well and has good language skills. In fact, when she started preschool at age 3, her fine motor skills were so good that she didn’t qualify for special services in that area.
Looking at Mia, most people would never suspect she spends every other Friday at Hasbro Children’s...